In a recent study published in the Nature Biomedical Engineering, a group of researchers optimized prime editing (PE) for the efficient correction of the cystic fibrosis (CF) transmembrane conductance ...

The Phase 2 randomized, double-blind, placebo-controlled study treated 128 people with CF ages 18 and older with two copies of the F508del mutation. One group of patients was randomized to receive ...

An optimized version of prime editing technology raises the possibility of a one-time treatment for cystic fibrosis. In their 2019 paper, Liu’s team used prime editing to alter the gene mutations ...

- With this reimbursement agreement in place, more than 2,200 people with CF will have PBS-funded access to TRIKAFTA®, including more than 700 who will now have access to a CFTR modulator therapy for ...

Vertex Pharmaceuticals (VRTX) announced the U.S. Food and Drug Administration has approved the expanded use of Trikafta for the treatment of people with cystic fibrosis ages 2 and older who have at ...

The Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) for vanzacaftor/tezacaftor/deutivacaftor triple combination ...